CLINICAL TRIALS PROFILE FOR METYROSINE

All Clinical Trials for metyrosine

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03512756 ↗	A Randomized Phase 2/3 Multi-Center Study of SM-88 in Patients With Metastatic Pancreatic Cancer	Active, not recruiting	Tyme, Inc	Phase 2/Phase 3	2018-03-27	A prospective, open-label phase 2/3 trial in metastatic pancreatic cancer subjects who have failed two lines of prior systemic therapy. The trial is designed to evaluate the safety and efficacy of SM-88 used with MPS (methoxsalen, phenytoin and sirolimus) in pancreatic cancer and will measure multiple endpoints, including overall survival, progression free survival, relevant biomarkers, quality of life, safety, and overall response rate. (Part 1 enrollment complete) In the initial stage of the trial (36 subjects), two dose levels of SM-88's metyrosine-derivative was evaluated. (Part 2 actively enrolling) The second part will consist of a subsequent expansion of the trial to further assess safety and efficacy of SM-88 used with MPS containing the selected SM-88 RP2D from Part 1. A total of 250 subjects in the second part will be randomized 1:1 either to the SM-88 arm (125 subjects) or Physician's Choice of therapy for the Control Arm (125 subjects). Subjects should have previously received two lines of prior systemic therapy.
NCT01127503 ↗	Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome	Terminated	Bausch Health Americas, Inc.	Phase 2	2010-06-01	This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).
NCT01127503 ↗	Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome	Terminated	Valeant Pharmaceuticals International, Inc.	Phase 2	2010-06-01	This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with velocardiofacial syndrome (VCFS).
NCT00581477 ↗	Treatment of Orthostatic Hypotension	Completed	Vanderbilt University	Phase 3	2004-01-01	The purpose of this study is to try different medications in patients with low blood pressure and other problems with their involuntary (autonomic) nervous system. The pharmacological trials in this study will perhaps lead to more effective treatment. This study consists of single dose trials, dose selection trials, 5-day trials and chronic (approximately 2 months) trials.
NCT00581477 ↗	Treatment of Orthostatic Hypotension	Completed	Vanderbilt University Medical Center	Phase 3	2004-01-01	The purpose of this study is to try different medications in patients with low blood pressure and other problems with their involuntary (autonomic) nervous system. The pharmacological trials in this study will perhaps lead to more effective treatment. This study consists of single dose trials, dose selection trials, 5-day trials and chronic (approximately 2 months) trials.

Clinical Trial Conditions for metyrosine

Condition Name

Condition Name for metyrosine
Intervention	Trials
Sarcoma, Ewing	1
Velo-cardio-facial Syndrome	1
Autonomic Nervous System Diseases	1
Dopamine Beta-Hydroxylase Deficiency	1

Condition MeSH

Condition MeSH for metyrosine
Intervention	Trials
Facial Paralysis	1
Sarcoma	1
Autonomic Nervous System Diseases	1
DiGeorge Syndrome	1

Clinical Trial Locations for metyrosine

Trials by Country

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−

Trials by Country for metyrosine
Location	Trials
United States	19

Trials by US State

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−

Trials by US State for metyrosine
Location	Trials
California	2
New York	2
Iowa	1
Illinois	1
Florida	1

Clinical Trial Progress for metyrosine

Clinical Trial Phase

Clinical Trial Phase for metyrosine
Clinical Trial Phase	Trials
Phase 3	1
Phase 2/Phase 3	1
Phase 2	2

Clinical Trial Status

Clinical Trial Status for metyrosine
Clinical Trial Phase	Trials
Terminated	1
Active, not recruiting	1
Completed	1

Clinical Trial Sponsors for metyrosine

Sponsor Name

Sponsor Name for metyrosine
Sponsor	Trials
Tyme, Inc	2
Vanderbilt University	1
Vanderbilt University Medical Center	1

Sponsor Type

Sponsor Type for metyrosine
Sponsor	Trials
Other	4
Industry	4

Metyrosine: A Comprehensive Update on Clinical Trials, Market Analysis, and Projections

Introduction to Metyrosine

Metyrosine, a small molecule drug, is primarily used in the treatment of pheochromocytoma, a rare tumor of the adrenal glands that leads to excessive production of adrenaline and noradrenaline. Developed by Merck Sharp & Dohme Research Laboratories, metyrosine was first approved in the United States in October 1979[1].

Mechanism of Action

Metyrosine works by inhibiting the enzyme tyrosine hydroxylase (TYH), which is crucial for the production of neurotransmitters such as dopamine, norepinephrine, and epinephrine. By reducing the activity of TYH, metyrosine decreases the production of these neurotransmitters, thereby helping to manage the symptoms associated with pheochromocytoma[1].

Clinical Trials and Approval Status

Metyrosine has undergone rigorous clinical trials and has been approved for use in several countries. The drug's approval highlights its efficacy and safety profile. However, there is limited recent data on ongoing clinical trials specifically for metyrosine, as it has been on the market for several decades.

Historical Context

The approval of metyrosine in 1979 marked a significant milestone in the treatment of pheochromocytoma. Since then, it has been prescribed to patients to help control the symptoms of this rare tumor. While there are no recent clinical trials focused solely on metyrosine, its continued use underscores its therapeutic benefits and minimal side effects[1].

Market Analysis

Current Market Scenario

The market for drugs targeting rare tumors like pheochromocytoma is niche but critical. Metyrosine, being one of the few approved treatments for this condition, holds a significant position in this market segment.

Growth Drivers

• Increasing Prevalence of Chronic Diseases: While not directly related to pheochromocytoma, the overall trend of increasing chronic diseases drives investment in pharmaceutical R&D, which can indirectly benefit drugs like metyrosine through advancements in related therapeutic areas[2][3].
• Advancements in Biotechnology: The growing emphasis on biotechnology and personalized medicine can lead to better diagnostic tools and treatments for rare conditions, potentially increasing the demand for targeted therapies like metyrosine[2].

Market Size and Projections

While specific market size data for metyrosine is not readily available, the broader market for rare disease treatments is expected to grow. For instance, the oral proteins and peptides market, which includes various therapeutic agents, is projected to grow from $1.27 billion in 2024 to $2.86 billion by 2028 at a CAGR of 22.4%[2].

Regional Breakdown

The global clinical trials market, which includes trials for various diseases, shows significant regional variations. North America and Europe are major players, but the Asia Pacific region is expected to see the fastest growth due to the increasing prevalence of chronic and infectious diseases[3].

Competitive Landscape

Other TYH Inhibitors

Metyrosine is part of a class of drugs known as TYH inhibitors. While it is one of the earliest and most established drugs in this class, other TYH inhibitors may be in various stages of development. The competitive landscape is influenced by the presence of these newer drugs and their potential to offer improved efficacy or safety profiles[1].

Future Trends and Projections

Biopharma Trends

Several trends in the biopharma industry are likely to impact the market for drugs like metyrosine:

• Increased Focus on Personalized Medicine: The growing emphasis on personalized medicine could lead to more targeted therapies, potentially increasing the demand for drugs that address specific rare conditions[5].
• Advancements in Gene Therapy: While not directly related to metyrosine, advancements in gene therapy, as seen in trials for conditions like Rett syndrome, indicate a broader trend towards more precise and effective treatments. This could influence the development of future treatments for pheochromocytoma[4].

Regulatory and Financial Factors

• VC Deals and IPOs: The biopharma sector is seeing a resurgence in venture capital deals and IPOs, which can provide the necessary funding for R&D and clinical trials. This financial activity can support the development of new treatments and potentially impact the market for existing drugs like metyrosine[5].

Key Takeaways

• Metyrosine's Established Role: Metyrosine remains a crucial treatment for pheochromocytoma, with a well-established safety and efficacy profile.
• Market Growth Drivers: The market for rare disease treatments is driven by increasing chronic disease prevalence, advancements in biotechnology, and a growing emphasis on personalized medicine.
• Regional Market Dynamics: The Asia Pacific region is expected to see significant growth in the clinical trials market, driven by the increasing prevalence of chronic and infectious diseases.
• Future Trends: The biopharma industry's focus on personalized medicine and advancements in gene therapy could influence the development of future treatments for rare conditions.

FAQs

What is metyrosine used for?

Metyrosine is primarily used in the treatment of pheochromocytoma, a rare tumor of the adrenal glands that causes excessive production of adrenaline and noradrenaline[1].

How does metyrosine work?

Metyrosine works by inhibiting the enzyme tyrosine hydroxylase (TYH), which is involved in the production of neurotransmitters such as dopamine, norepinephrine, and epinephrine[1].

When was metyrosine approved?

Metyrosine was first approved for use in the United States in October 1979[1].

What are the current market trends affecting metyrosine?

The market for metyrosine is influenced by trends such as the increasing prevalence of chronic diseases, advancements in biotechnology, and a growing emphasis on personalized medicine[2][3].

Are there any new clinical trials for metyrosine?

There is limited recent data on ongoing clinical trials specifically for metyrosine, as it has been on the market for several decades. However, the broader biopharma industry is seeing significant activity in clinical trials for various diseases[3].

How does the competitive landscape affect metyrosine?

Metyrosine competes with other TYH inhibitors and potential new treatments that may offer improved efficacy or safety profiles. The competitive landscape is dynamic and influenced by advancements in biotechnology and personalized medicine[1][5].

Sources

1. Decoding Metyrosine: A Comprehensive Study of its R&D Trends - Synapse by Patsnap
2. Oral Proteins and Peptides Market Report 2025 - GlobeNewswire
3. Clinical Trials Market SIZE, SHARE | GROWTH REPORT [2032] - Fortune Business Insights
4. Rett Research & Clinical Pipeline - International Rett Syndrome Foundation
5. Seven Biopharma Trends to Watch in 2025 - Genetic Engineering & Biotechnology News